UMBILICAL CORD BLOOD BANK FOR HEMOGLOBINOPATHY PATIENTS

在过去的十年中，在以下方面取得了一些进展 治疗或治疗镰状细胞性贫血和地中海贫血。其中包括 异基因骨髓移植是目前唯一的治疗方法 这提供了治愈的可能性。在镰状细胞性贫血中， 移植已经在晚期患者身上进行过。 地中海贫血时的器官损害，已经进行了移植 在发现铁相关组织损伤的证据之前。由于对……的担忧 移植和移植物抗宿主病(GVHD)，移植治疗 患有血红蛋白疾病的患者仅限于 存在与人类白细胞抗原相合的供者。不幸的是，一名人类白细胞抗原匹配的亲属 经常找不到捐赠者。脐带血，最近一次 公认的造血干细胞来源，已被用于 成功移植了500多名患者。的潜在优势 脐带血高于其他干细胞捐赠者来源与 观察到，即使没有完全的人类白细胞抗原相容，风险 高级别GVHD是最低限度的。这项建议的目的是 增加提供移植作为治疗选择的机会 通过建立一个与血红蛋白疾病相关的基因 脐带血库。我们的具体目标是(1)建立一个 脐带血库，包含2500个脐带血单位(CBU)，将提供 作为血红蛋白治疗中心的资源，(2)实施、验证 并监测这些中心的CBU收集程序，(3)确定 如果可以在多个地点收集高质量的CBU并将其运往 儿童医院奥克兰研究所(CHORI)进行处理 和冷冻保存，以及(4)建立研究资源 CBU移植在慢性粒细胞白血病患者中的作用评价 血红蛋白病。脐带血采集、加工和采集协议 数据收集改编自NIH无关的脐带血 移植研究(COBLT)。统计评价和计算机化 数据收集将由Emmes，Corp.执行，该组织提供 对COBLT研究的类似援助。人类白细胞抗原分子配型与临床 将向以下人员提供血红蛋白病诊断服务 协作计划。中描述的相关CBU资源 申请将为调查人员提供一个研究 CBU在血红蛋白病患者移植中的作用 在分子水平上评估人类白细胞抗原差异， 使用Cbus植入和GVHD，并确定Cbus是否收集 应该提供给所有家庭，如果他们的孩子有一个 或有可能生下一个患有 血红蛋白病。这个相关的脐带血库提供的资源 可能会为一些患者提供治愈的可能性 受镰状细胞性贫血和地中海贫血的影响。

During the past decade, a number of advances have been made in the treatment or patients sickle cell anemia and thalassemia. Among these is allogeneic bone marrow transplantation, the only current treatment that offers the potential for cure. In sickle cell anemia, transplantation has been performed in patients who have had advanced organ damage while in thalassemia, transplantation has been performed before evidence of iron related tissue damage. Due to concerns over engraftment and graft versus host disease (GVHD), transplants for patients with hemoglobinopathies have been limited to situations where an HLA compatible donor exists. Unfortunately, an HLA matched related donor is often not available. Umbilical cord blood, a recently recognized source of hematopoetic stem cells, has been used to successfully transplant over 500 patients. The potential advantages of cord blood over other donor sources of stem cells is related to the observation that even without complete HLA compatibility, the risk of high-grade GVHD is minimal. The objective of this proposal is to increase the chance to offer transplantation as a therapeutic option for patients with hemoglobinopathies by establishing a genetically related umbilical cord blood bank. Our specific aims are (1) to establish a cord blood bank containing 2500 cord blood units (CBU) which will serve as a resource to hemoglobinopathy centers, (2) to implement, validate and monitor CBU collection procedures in these centers, (3) to determine if quality CBUs can be collected at multiple sites and shipped to Children's Hospital Oakland Research Institute (CHORI) for processing and cryopreservation, and (4) to establish a research resource for evaluation of the role of CBU transplantation in patients with hemoglobinopathies. Protocols for cord blood collection, processing and data collection have been adapted from the NIH unrelated Cord Blood Transplantation study (COBLT). Statistical evaluation and computerized data collection will be performed by The EMMES, Corp., a group providing similar assistance to the COBLT study. Molecular HLA typing and hemoglobinopathy diagnostic services will be made available to collaborating programs. The related CBU resource described in this application will provide investigators with an opportunity to study the role of CBU for transplantation in patients with hemoglobinopathies, to evaluate at the molecular level relationships between HLA disparity, engraftment and GVHD using CBUs, and to determine if CBUs collection should be offered to all families who either have a child with a hemoglobinopathy or are at risk of having a child with a hemoglobinopathy. The resource provided by this related cord blood bank may offer the possibility of cure to a number of patients who are affected by sickle cell anemia and thalassemia.